MPNforum Magazine

 A fresh approach to MF research

The news flashed over the wires two weeks ago.  It was followed by massive e-mails to MPN patients:   MPN Research Foundation and the Leukemia & Lymphoma Society are partnering to fund the Myelofibrosis Challenge, a project to stimulate innovation in fibrosis research,. The collaboration of MPNRF and LLS  is just the latest development in MPN research that is suddenly heating up.  In part because of  Jakafi,  a  JAK inhibitor getting FDA approval for myelofibrosis, there has been a tsunami of publishing in the  MPN scientific space. “It’s the story of the year,” says Dr. Isabel Cunningham in OncoStat, “More than 2000 articles published and over 3000 ASH abstracts this past year”. Why the excitement?  “It’s Sutton’s Law,” says Dr. Cunningham. “Research goes where the money is,” More money is going into this MF Challenge and small wonder.  Digging just a little past the headlines the bold and innovative nature of this new joint initiative begins to emerge. It’s not even two heavyweight foundations joining forces to fight MPNs…although that would be reason enough. And it’s not only that the focus of the collaboration is fibrosis research, focusing on a tissue consequence of MF  rather than the genetic causes….although that paradigm shift might be the source of  the MF Challenge’s biggest payoff. It’s the very nature of the “spur to innovation” deployed by MPNRF and LLS that has generated widespread excitement.   These are concept grants, wide open to big established labs and brilliant post docs working out of their grad school apartments, open to specialists in and out of genetics, hematology or other disease specific disciplines.  The idea is to fish for fresh approaches to the essential pathogenesis of MPNs using the widest possible net.The MF Challenge concept grants, funded up to $100,000 each, will be awarded based on written proposals,

Barbara Van Husen,  Robert Rosen, the MPN Research Foundation

scientific board review, and personal interviews.  The deadline for proposals is six weeks from now,  April 1, 2012 (see below for schedule and application info.) The MF Challenge: Officially, according to the MPN Research Foundation and the Leukemia & Lymphoma Society Request for Proposals:  “The goal of the MF Challenge is to discover the factor (s) that induce(s) fibrosis in bone marrow and to identify opportunities to arrest and reverse t his fibrosis.   We believe that such a campaign will be a game-changing opportunity for MF science and a life-changing opportunity for MF patients.” MyelofibrosisBeyond standing with the most life-threatening of MPNs, myelofibrosis is an acute end point of a continuum that starts with chronic ET and PV and moves on to MF and the acute leukemias.  And yet, for all the major advances in genetic understanding and new drug development,  little research has been done into causes of fibrosis in the marrow cavity. Although responsible for suffering and morbidity, the phenomenon of fibrosis in the MPN phenotype has been regarded as a result of proliferating blood lines rather than a subject of research on its own account.  Researchers have generally  focused their efforts on discovering mutations causing the pathogensis in order to isolate an effective target for drug development.

“We have never,” says Barbara Van Husen, president of MPN Research foundation, “received a request for funding based purely on research into fibrosis.  We are going to change that with this program.“

Now that the idea is on the table – attack and reverse the clear impact of myelofibrosis —  the fibrosis itself, it all seems so inevitable. Here’s how it started: “The idea of focusing on fibrosis,” said Robert Rosen, MPNRF CEO, ” came from a patient, one of our donors.   We took it to our scientific advisory board and were surprised and delighted that they concluded it was a good idea for right now since advances in fibrosis research for other organs has not been brought to bear on bone marrow fibrosis. “We took it on the road and talked with scientists we rely on for feedback,  Andy Shafer from Weil Cornell, head of our scientific advisory board and to others in the community.We had conference calls through the summer, then talked it over with John Crispino at Northwestern to develop a White Paper. “Finally, at the board meeting in New York in November, it all came together and we started looking for partners. That’s when we moved into advanced talks with Lou DeGennaro, Rick Winneker and the folks at the Leukemia & Lymphoma Society.  We became partners in this project and are jointly financing the concept grants.” LLS is no stranger to the MPN world.  The Society currently has a total of  nine grants that include MPN research .   The dollar commitment for those grants this year is approximately $2.7 million.  Beyond that,  the Leukemia & Lymphoma Society  offers free information and patient services for individuals and families touched by blood cancers, including MPNs. Richard Winneker,PhD.,   senior vice president  of research at the Leukemia & Lymphoma Society, came to LLS two years ago with a 20 year  drug discovery and development background, mainly as a research executive at Wyeth Research and also as an independent consultant. “When Bob Rosen and Barbara Van Husen contacted  us,” said Winneker,  “they were assessing the program and wanted to explore our participation. This is something that is competely consistent with our mission. Their innovative scientific approach really spoke to us.  Since 1954 we’ve supported extraordinary academic research and that’s still one of our main objectives.  However, we’ve been implementing new planned funding mechanisms as well to fill some important gaps that don’t fall within our traditional grant award parameters.    This fits nicely in that space.   Last year LLS provided over $76 million in academic and biotech related research awards and partnerships.”  The concept grants  “The way we’re doing this project, “said Van Husen, “is also innovative. One of our science advisers said many of the easy targets for MF have been investigated without a lot to show for it.  We need new ideas, new concepts,. “When we were considering the MF Challenge providing  a concept grant — — funding initial exploration of untested but potentially transformative research ideas —  instead of granting funds for a specific research project seemed the most likely path to explore fresh ideas.

By reaching into the academic word, into industry and  the larger fibrosis world –pulmonary fibrosis,  renal interstitial fibrosis, and systemic sclerosis —  and having contact with those scientific boards, we’re hoping  to find interesting ideas to move forward into formal research.

“We should  attract people looking less at body parts than molecular structure and signaling pathways that give rise to fibrosis.  So much work has already been done in other disease areas that just lies fallow for lack of application.  This might also be one path to find application to MF of earlier discoveries in unrelated diseases.” In case you want to apply  Concept grants are on a fast track.  Here’s the way it will work.  The deadline for the MF Challenge grant proposals is April 1.  As proposals arrive, they are scored.   On June 1,  final review starts in Chicago.   Representatives of LLS and MPNRF as well as national and international fibrosis scientists will gather around a table in Chicago and review every scored  proposal. (ASCO will be in Chicago that weekend, so there many will be there in any case.) After those discussions, panel members have the opportunity to adjust their scores, if they wish.  MPNRF and LLS will meet to determine the final award of four $100,000 concept grants. (“If there are five compelling proposals,” says Van Husen, “we’ll scramble to find the find the money”) Since all this takes place against the background of MPNRF’s on-going efforts to support continuing and new projects it’s going to be a hot time in Chicago this summer. 

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© Zhenya Senyak and MPNforum.com, 2012. Unauthorized use and/or duplication of this material without express and written permission from this blog’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Zhenya Senyak and MPNforum.com with appropriate and specific direction to the original content.